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Reclutamiento
ID del estudio de Astellas
0367-CL-1201

A study to evaluate ASP0367 in participants with primary mitochondrial myopathy

ID del estudio de Astellas
0367-CL-1201
Id. de ClinicalTrials.gov
NCT04641962
EUCTIS ID
N/A
Id. de EudraCT
N/A
jRCT ID
N/A
CN CDE ID
N/A
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Resumen del estudio

The purpose of the phase 2 portion of this study is to select a biologically-active ASP0367 dose level by pharmacokinetic (PK) and pharmacodynamic (PD) evaluation. The phase 2 portion of this study will also assess the safety and tolerability of ASP0367.

The purpose of the phase 3 portion of this study is to assess the effect of ASP0367 on functional improvement relative to placebo and will also assess the safety and tolerability of ASP0367 relative to placebo. The phase 3 portion of this study will also assess the effect of ASP0367 on functional improvement and fatigue relative to placebo and will assess the effect of ASP0367 in overall participant functioning relative to placebo.

Más información del estudio

Fase
Phase 2/3
Producto
ASP0367
Producto
  • ASP0367
  • Placebo
    • Tipo
    Interventional
    Enmascaramiento
    Triple (Participant, Care Provider, Investigator)
    Número de inscripción
    149
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    Obtenga más información

    ¿Le gustaría obtener más información sobre los centros del estudio clínico que están reclutando a participantes para el estudio A study to evaluate ASP0367 in participants with primary mitochondrial myopathy? Comuníquese con nosotros completando sus datos a la derecha y le responderemos.

    • A caregiver
    • A healthcare provider
    • A parent
    • A patient
    • A patient advocate
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    Al hacer clic en [“Continuar/Enviar”], usted acepta que Astellas se comunique con usted por correo electrónico con información que lo ayude a conectarse con los centros de estudios clínicos. Si bien podemos conectarlo con los centros de estudios clínicos, no podemos responder preguntas sobre ningún tratamiento en investigación por correo electrónico. Su consentimiento para recibir correos electrónicos no es un requisito para participar en un estudio o estudio clínico. Para obtener más información, incluida la forma de cancelar la suscripción en cualquier momento, consulte nuestro Aviso de privacidad y política de cookies.

      Ubicaciones

      Contáctenos
      Contáctenos
      Reclutamiento
      Akron Children’s Hospital
      Akron, Ohio, Estados Unidos, 44308
      Contáctenos
      Información del estudio
      Estudio de seguimiento
      Edad
      18 - 80 years
      Diamond icon
      Afección
    • Miopatía mitocondrial primaria (MMP)
      • Gender icon
      Sexo
      Mujer y hombre
      Calendar icon
      Fechas del estudio
      May 2021 - Oct 2023
      Más información del estudio
      Criterios de inclusión
      • Participant agrees and is able to adhere to the study requirements for the length of the study, including performing 6MWT, as well as the use of digital applications and video recordings.
      • Diagnosed with primary mitochondrial myopathy (PMM), consisting of the following:
        • Molecular genetic abnormality (i.e., nuclear or mitochondrial) known to be associated with causing mitochondrial dysfunction (such as, but not limited to, mitochondrial DNA (mtDNA) single, variable deletions in chronic progressive external ophthalmoplegia (CPEO) and Kearns-Sayre syndrome (KSS); mtDNA m.3243 A > G common mutation in mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes (MELAS); pathogenic nuclear or mitochondrial genome variants demonstrated to cause primary mitochondrial disease), and
        • Participant reported symptoms (i.e., muscle weakness, fatigue and exercise intolerance) or physical examination findings of myopathy that are the predominant symptoms of the participant’s mitochondrial disorder.
      • Participant has been on stable dose regimen of coenzyme Q10 (CoQ10), carnitine, creatine or other mitochondrial disease-focused vitamins or supplemental therapies for 3 months prior to randomization and intends to stay on a stable dose for duration of study period (for participants who take any above-mentioned medications or supplements).
      • Participant has been on stable exercise regimen within 4 weeks prior to randomization and intends to stay on a stable regimen for duration of study period (for participants who participate in a regular exercise regimen).
      • La participante de sexo femenino no está embarazada y se aplica por lo menos una de las siguientes condiciones:
        • No es una mujer en edad fértil (MEF).
        • WOCBP who agrees to follow the contraceptive guidance from the time of informed consent through at least 30 days after final study treatment administration.
      • Female participant must agree not to breastfeed starting at screening and throughout the study period and for 30 days after final study treatment administration.
      • Female participant must not donate ova starting at first dose of IP and throughout the study period and for 30 days after final study treatment administration.
      • Male participant with female partner(s) of childbearing potential (including breastfeeding partner) must agree to use contraception throughout the treatment period and for 30 days after final study treatment administration.
      • Male participant must not donate sperm during the treatment period and for 30 days after final study treatment administration.
      • Male participant with pregnant partner(s) must agree to remain abstinent or use a condom for the duration of the pregnancy throughout the study period and for 30 days after final study treatment administration.
      • La participante acepta no participar en otro estudio intervencionista mientras forma parte del presente estudio.

        • Open-label Extension Continuation Criteria:

      • Participant must meet all of the following OLE criteria at the week 52 study visit in the treatment period to be eligible for OLE:
        • Participant must continue to be able and willing to adhere to the study requirements.
        • Participant who is eligible to continue in OLE.
      MaximizeCriterios de exclusión
      • Participant has additional signs and/or symptoms due to non-myopathic process (e.g., cerebellar dysfunctions, movement disorder, peripheral neuropathy, stroke or other) or a gait problem not attributed to the myopathy that would interfere/may in addition to the myopathy affect the participant’s performance during 6MWT or 5 times sit to stand (5XSTS).
      • La participante ha recibido cualquier terapia de investigación dentro de los 28 días o 5 semividas, el período que sea más largo, antes de la selección.
      • Participant has any condition, which makes the participant unsuitable for study participation.
      • Participant has cardiac troponin I (cTnI) above the upper limit of normal (ULN) (or cardiac troponin T [cTnT] above the ULN if cTnI is not available) at screening.
      • Participant has estimated glomerular filtration rate (eGFR) calculated by the Modification of Diet in Renal Disease equation < 60 mL/min/1.73 m^2 at screening.
      • Participant has at screening: total bilirubin (TBL) > ULN or transaminase(s) (aspartate aminotransferase [AST] or alanine aminotransferase [ALT]) > ULN in the absence of elevations in creatine kinase (CK).
      • Participant has psychiatric conditions such as schizophrenia, bipolar disorder or major depressive disorder that has not been under control within 3 months prior to screening.
      • Participant has a history of suicide attempt, suicidal behavior or has any suicidal ideation within 1 year prior to screening that meets criteria at a level of 4 or 5 by using the Columbia Suicide Severity Rating Scale (C-SSRS) or who is at significant risk to commit suicide.
      • Participant has severe behavioral or cognitive problems that preclude participation in the study.
      • Participant has undergone an in-patient hospitalization that precludes participation in the study, within the 30 days prior to the randomization.
      • Participant has a planned hospitalization or a surgical procedure during the study, which may affect the study assessments.
      • Participant has clinically significant and unstable respiratory disease and/or cardiac disease (medical history or current clinical findings), or prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to randomization.
      • Participant has a corrected mean QT interval using Fridericia’s correction (QTcF) > 450 msec for male participants and > 480 msec for female participants at screening or randomization. If QTcF exceeds these limits, one additional triplicate ECG can be repeated on the same day in order to determine the participant’s eligibility.
      • ECG evidence of acute ischemia, atrial fibrillation or active conduction system abnormalities with the exception of any of the following:
        • First degree atrioventricular (AV)-block
        • Second degree AV-block Type 1 (Mobitz Type 1/Wenckebach type)
        • Right bundle branch block
        • Left fascicular block
        • Bi-fascicular block
      • Participant requires 24/7 ventilator support (those who require nocturnal ventilation support such continuous positive airway pressure [CPAP] or bilevel positive airway pressure [BiPAP], due to nighttime hypoxia from chest muscle weakness or obstructive sleep apnea are allowed).
      • Participant has severe vision impairment that may interfere with their ability to complete all study requirements.
      • Participant has an intractable seizure disorder that may interfere with their ability to complete all study requirements.
      • Active malignancy or any other cancer from which the participant has been disease-free for < 5 years, except for curative treated localized non-melanoma skin cancer (e.g., basal cell or squamous cell carcinoma).
      • Participant has a solid organ transplant and/or is currently receiving treatment with therapy for immunosuppression.
      • Participant has severe scoliosis or kyphoscoliosis that significantly impair respiratory capacity and pulmonary function tests or limit positioning due to pain whom would be likely to require orthopedic surgical intervention within a year after study randomization.
      • Participant has a positive for human immunodeficiency virus (HIV), hepatitis B or hepatitis C infection at screening.
      • Participant has previously received ASP0367.
      • Participant has a history of active substance abuse within 1 year prior to randomization.
      • Participant has used any peroxisome proliferator-activated receptors (PPAR) ligands such as fibrates and thiazolidinediones within 4 weeks prior to randomization.
      • Participant has initiated the use of CoQ10, carnitine, creatine or other mitochondrial disease-focused supplements within 3 months prior to study randomization.
      • Participant has a known or suspected hypersensitivity to ASP0367 or any components of the formulation used.

      Preguntas frecuentes

      ¿Los estudios clínicos son solo para personas con cáncer avanzado?

      Si bien algunos estudios clínicos pueden enfocarse en cánceres más avanzados, muchos estudios están abiertos para pacientes con cáncer en diferentes etapas. Cada estudio tiene reglas que establecen quiénes pueden participar. Por ejemplo, quizás solo puedan participar pacientes de un grupo de edad determinado o aquellos que tengan un tipo determinado de tumor.

      ¿Será necesario que interrumpa mi tratamiento actual para participar en un estudio clínico?

      A veces los investigadores quieren que los participantes sigan recibiendo sus tratamientos actuales durante un estudio clínico. En otras ocasiones, es posible que deba interrumpir sus tratamientos actuales durante un tiempo. Si el tratamiento en investigación no funciona, siempre puede volver a su plan de tratamiento original.

      ¿Debería preocuparme la posibilidad de recibir placebo?

      En los estudios clínicos oncológicos, solo se usa placebo si no hay otro tratamiento disponible para ese tipo de cáncer. Esto ayuda a comparar un tratamiento en investigación con el placebo. Los placebos rara vez se usan en estudios oncológicos, porque en su lugar generalmente se administra el mejor tratamiento disponible, que se denomina “estándar de atención”.

      ¿Necesito una derivación de mi médico para participar en un estudio?

      Es posible que su médico no conozca todas las oportunidades de participación en estudios clínicos que usted tiene a su disposición. Hable con su médico o con otro profesional de la salud sobre la información que encuentre en relación con un estudio clínico. Podrán ayudarlo a decidir si el estudio clínico es adecuado para usted. Si no encuentra ninguna opción en este sitio web, le recomendamos que visite un registro público en línea, por ejemplo, clinicaltrials.gov para conocer los diversos estudios clínicos que están disponibles.

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